Boehringer Ingelheim Exercises Option on Second Hepatic Disease Target From Research Collaboration with Dicerna

INGELHEIM, Germany & CAMBRIDGE, Mass., January 4, 2019

Boehringer Ingelheim and Dicerna Pharmaceuticals (NASDAQ: DRNA), a leading developer of investigational RNA interference (RNAi) therapeutics, today announced that Boehringer Ingelheim has exercised an option to receive exclusive rights to a second hepatic disease target emerging from its research collaboration and license agreement with Dicerna. The collaboration, established in October 2017, aims to discover and develop novel GalXC™ RNAi therapeutics for the treatment of chronic liver diseases, with an initial focus on nonalcoholic steatohepatitis (NASH), a devastating disease for which there is no approved treatment.

The option is the second target under the two companies’ research collaboration and license agreement. Under the terms of the agreement, Boehringer Ingelheim will be responsible for future clinical development and commercialization of the therapeutic target. Dicerna is eligible to receive development and commercial milestone payments, and royalties on worldwide net sales.

“It is extremely gratifying for Boehringer Ingelheim to exercise its option for a second therapeutic candidate from our productive research collaboration,” said Douglas M. Fambrough, President and Chief Executive Officer of Dicerna. “The option exercise underscores the potential of Dicerna’s GalXC technology platform in facilitating the discovery and development of novel RNAi therapies for nonalcoholic steatohepatitis and other chronic liver diseases. We look forward to continued progress and the potential advancement of clinical candidates from our partnership.”

Dicerna and Boehringer Ingelheim selected the target based on its ability to be drugged using Dicerna’s proprietary GalXC technology platform. The GalXC platform uses RNAi to inhibit the expression of disease-causing genes by destroying the messenger RNAs (mRNAs) of those genes. The approach has the potential to treat diseases by silencing previously inaccessible drug targets.

“Boehringer Ingelheim is pleased with the progress of our initial collaboration with Dicerna and based on early findings and our strong relationship, we are expanding our research efforts,” said Clive R. Wood, Ph.D., Corporate Senior Vice President Discovery Research at Boehringer Ingelheim. “Today’s announcement exemplifies our commitment to discovering new therapies for NASH and other chronic liver diseases and advances our research efforts in this priority area.”

NASH is caused by the buildup of fat in the liver, potentially leading to liver fibrosis and cirrhosis. It has an especially high prevalence among obese and diabetic patients and is an area of high unmet medical need. NASH is expected to soon become the most common cause of advanced liver disorders, and it often necessitates liver transplantation.

Dicerna is building a portfolio of research and development programs to advance the treatment of diseases involving the liver, including chronic liver diseases, viral infectious diseases, cardiovascular diseases and rare diseases. The company aims to leverage its proprietary GalXC technology to develop innovative RNAi therapeutics to positively transform the lives of individuals living with these diseases.

Cautionary Note on Forward-Looking Statements

This press release includes forward-looking statements. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Examples of forward-looking statements include, among others, statements Dicerna makes regarding: (i) the therapeutic and commercial potential of GalXC™ (ii) research and development plans related to GalXC; and (iii) the potential of our technology and drug candidates in our research and development pipeline. The process by which an early stage platform such as GalXC could potentially lead to an approved product is long and subject to highly significant risks, particularly with respect to a pre-clinical research collaboration. Applicable risks and uncertainties include those relating to Dicerna’s preclinical research and other risks identified under the heading Risk Factors included in Dicerna’s most recent Form 10-Q filing and in other future filings with the SEC. The forward-looking statements contained in this press release reflect Dicerna's current views with respect to future events, and Dicerna does not undertake and specifically disclaims any obligation to update any forward-looking statements.